Saturday, 4 July 2015

Drawing breath

The last few days have seen
some dramatic breaking news for the cystic fibrosis community, with results
from the gene therapy Phase 2b clinical trial being published, NHS England
announcing funding for ivacaftor for eight rare mutations, and the FDA in the US licensing the combination treatment Orkambi.
In this blog, Trust Chief Executive Ed Owen looks back on a momentous couple of days, and how the power
of the CF community shines through.

The last 48 hours
has proved a significant step forward in our fight for a life unlimited by
cystic fibrosis – and one that reinforces the collective power we have as a
community, both national and global.

On Friday morning
the results of the gene therapy Phase 2b clinical trial were published in the Lancet Respiratory journal.
While not a surprise to those who took part in our sneak preview a few weeks ago, the outcome is
certainly encouraging.

More clinical
trials are needed before we can say gene therapy can be a viable treatment for
people with cystic fibrosis, but it successfully establishes proof of concept
that demonstrates genuine promise.

Future trials are
now needed and that will almost certainly require the sort of resources and
expertise that only a pharmaceutical company can provide. We also have to
be realistic and accept that this will take some time.

But it is good
news and one that has not come this far by accident.

First and
foremost, it is a tremendous tribute to Professor Eric Alton and the wider Gene
Therapy Consortium team for their ground-breaking work. Yet, as they would
be the first to say, this could not have happened without the participation of
people with cystic fibrosis, young and old, who have volunteered to take part
in this and earlier clinical trials.

And, vitally, it
has only come about because of the extraordinary support of the wider cystic
fibrosis community raising many millions of pounds over the last 15 years.

This is continuing
and the Trust has pledged a further £500,000
to support the GTC’s Wave 2 gene therapy product which – while about five years
behind the product that is the focus of this week’s news – has potentially much
greater impact.

The Trust will
continue to work tirelessly to ensure everyone with cystic fibrosis gets access
to life-changing therapies however long it takes. As with gene therapy and
other promising scientific development, that means investing funds raised in
research. But it also means lobbying and campaigning to ensure the NHS
funds therapies approved by regulators.

So we were
delighted with the news on Thursday that NHS England has agreed to extend access to Kalydeco
(ivacaftor) to about 40 people who have non-G551D gating mutations of cystic
fibrosis.

It has taken far
too long, and demonstrates the inadequate system in place to ensure such
transformational drugs are appraised. What’s more, a handful of people in
Wales are still waiting – and we will continue to fight for them until the
right decision is made.

But the voice of
people with cystic fibrosis supported by clinical teams and backed by clear
evidence of Kalydeco’s transformational impact rightly won the day.

There are many
more issues like this to come over the next years and a further big potential
campaigning issue on the horizon is Orkambi – the so-called combination therapy
eligible for those with two copies of the F508delta gene, the most common
mutation of cystic fibrosis.

Its effect is less
dramatic than that seen with Kalydeco on those with gating mutations. But
the evidence from Phase 3 trials
shows that it significantly reduces hospitalisations for those who take the
drug and stabilises lung function.

We are expecting a
decision from European regulators by the end of this year and, if approved, the
key issue will be whether it is reimbursed by the NHS. It is by no means
certain that it will, and we will be pushing Vertex and the NHS in the four
parts of the UK to do the right thing to enable Orkambi to get to those who
need it as soon as possible at an affordable price.

This week has shown that when we come
together as one we can change the world for many with cystic fibrosis. The
Power of Us will continue to be our key weapon to secure further changes vital
to our mission to transform the lives of all.